EXAPHARMA provides high quality source plasma that can be manufactured into lifesaving therapeutics, helping patients in Canada and around the world. Read about the different conditions that benefit from plasma derived therapeutics and success stories of patients who have used these products.
Clotting factor deficiencies
Factors VIII, IX and von willebrand factor (vWF) are crucial proteins involved in forming blood clots. They are essential in preventing excessive and uncontrollable bleeding. Some individuals are born with disorders where no or low levels of these proteins are produced in the blood. Under some instances such as excessive bleeding during surgeries and treatment with certain medications can lead to acquired clotting factor deficiencies
Factor VIII/IX concentrates and vWF factor can be extracted from source plasma and administered to these patients to alleviate the symptoms and deficiencies.
-Hemophilia A or Classical Hemophilia
Individuals born with hemophilia A produce no or reduced levels of factor VIII. Hemophilia A occurs almost always in males but can also affect females. One in every 10,000 individuals in Canada is born with Hemophilia A. To increase the levels of factor VIII in their blood individuals with hemophilia A are injected with plasma derived Factor VIII as part of the factor replacement therapy. -Hemophilia B (also known as the Christmas Disease)
Hemophilia B occurs due to no or reduced levels of factor IX in blood. Hemophilia B is shown to occur in 1 in every 50,000 individuals in Canada.
-Von Willebrand Disease
Individuals with Von Willebrand Disease (vWD) have very slow blood clotting and have recurrent nose bleeds and bleeding in the gums. vWD deficiency occurs in 1 in every 500 individuals in Canada. Individuals with vWF deficiency may also be deficient in Factor VIII.
Patients with the inherited bleeding disorders receive routine prophylactic treatment by injecting plasma derived Factor VIII, IX or vWF into their skin, muscle or vein. This treatment is known as factor replacement therapy. It takes 1200 donations per year to collect enough plasma to produce therapy for 1 patient with hemophilia A. Artificially produced factors by recombination have proven to be successful as an alternative treatment modality for patients with bleeding disorders.
-Excessive blood loss
Acquired deficiency of the clotting factors occurs in individuals undergoing surgeries with severe blood loss. Replacement of blood with red blood cell concentrates or volume expanders leads to further dilution of the clotting factors. Such patients are treated with plasma purified clotting factors to alleviate the acquired deficiency.
-Drug induced deficiency
Individuals treated with anticoagulants to prevent clotting in the veins (deep venous thrombosis) and organ damage can develop drug induced deficiency of the clotting factors. Some of the medications that can lead to clotting factor deficiency are unfractionated heparin, warfarin and enoxaparin. Patient with hemorrhages often treated with these anticoagulants bleed abnormally and produce clotting factor deficiency. Under such circumstances patients are administered clotting factors to promote haemostasis (process which causes bleeding to stop).
Other Clotting Factor Deficiencies:
There are a number of other factor deficiencies, some of which are hereditary and some acquired which are treated in a similar manner (as replacement therapy). These include deficiencies of factor I (Fibrinogen), II (Prothrombin), V, VII, X, XI and XIII.
Infections and immune deficiencies
The human immune system is designed to identify, attack and clear bacteria, viruses and other harmful foreign agents from our body. An essential component of the immune system that identifies and marks the foreign bodies for destruction are Immune globulins.
In some individuals however the immune globulins fail to make the distinction between foreign agents and body’s own cells and mark them for destruction leading to auto-immune diseases. In some instances, part or all components of the immune system are missing, making such individuals prone to infections.
Infusion with immune globulins from healthy donors is used as successful and effective treatment modality patients with immune deficiencies and certain auto-immune disorders.
-Idiopathic Thrombocytopenic Purpura (ITP):
Idiopathic Thrombocytopenic Purpura (ITP) is an auto-immune disease where the immune system attacks platelets leading to death of these cells. Platelets are cells that are essential for the formation of clots. Individuals with ITP bleed underneath their skin producing red or purple coloured rash (purpura) and have an increased tendency to bleed.
Patients with ITP are infused with immune globulin from healthy donors. The infused immune globulins block the ITP patient’s malfunctioning immune globulins, preventing the destruction of platelets. A rise in ITP patient’s platelet count begins to rise within a day or two and reaches normal levels within a week after the IVIG (intravenous immune globulin) infusion. However the therapeutic effect of the IVIG wanes over time and the patient must receive repeated infusions.
Kawasaki disease is an auto-immune disease characterized by inflammation of blood vessels throughout the body. One of the classical features of the disease includes erythema and edema of lips and tongue with a fever lasting more than five days. The cause of the Kawasaki disease is unknown and shown to occur most commonly in children under five years of age. The first-line treatment for this disease is IVIG infusion and upto 90% of patients respond promptly to this treatment modality.
Primary immunodeficiencies (PI) are congenital and are therefore most often diagnosed in children. There are over 150 types of PI and approximately 13,000 Canadians suffer from this disease. Common variable immunodeficiency (CVID) is a disease where very low levels of IgG and IgA immune globulins are produced by the body. IgA is a protein present in the mucosal membrane (respiratory, digestive, urogenital tract, etc.) and mucous secretions (saliva, tears, etc.) providing protection from microbial entry into the body. IgGs are by far the most critical and necessary proteins of the immune system that selectively bind and mark foreign agents for destruction. As a result individuals deficient in IgG and IgA can become infected very easily. Other types of PIs include severe combined immunodeficiency (SCID), selective immunoglobulin A deficiency, Wiscott-Aldrich syndrome and X-linked agammaglobulinemia. PI Patients deficient in immune globulins are infused with plasma derived immunoglobulins (IVIG) every three to four weeks, providing them with essential proteins required to fight infections.
-Secondary Immune deficiency:
Unlike the primary immune deficiencies the secondary immune deficiencies are acquired as a result of HIV/ AIDS infection, splenectomy, multiple myeloma, allogeneic stem cell and bone marrow transplantation, B-cell Chronic Lymphocytic Leukemia, and most chemotherapeutic agents that are immunosuppressive. Secondary immune deficiency is also observed in patients with diabetes mellitus (predisposition to fungal infections), rheumatoid arthritis, sickle cell anemia and many others. Patients with acquired immune deficiency are administered plasma derived immunoglobulins intravenously to gain immunity and to fight off infections.
-Acquired Passive Immunity:
When exposed to an infection the immune system mounts an immediate and generalized response against the foreign bodies. This primary immune response or innate immunity is however not very efficient at clearing infections. A more targeted response is initiated as part of the secondary immune response also known as adaptive immunity. White blood cells (B-cell lymphocytes) produce targeted proteins called immunoglobulins that identify, bind and mark the invading pathogen for destruction. It takes much longer (upto 14 days or longer) to mount this secondary immune response.
Plasma of individuals infected or vaccinated against a pathogen contains high titres of these targeted immunoglobulins which can be purified and collected. These immunoglobulins are known as hyperimmune globulins. Administration of hyperimmune globulins provides immediate and passive immunity to the recipient. Presently hyperimmune globulins exist for Hepatitis B, Rabies, Tetanus toxin, CMV, Rho D and many more.
Hyperimmune globulins are administered if a patient is immunodeficient and cannot mount an adequate immune response or if immediate post exposure prophylaxis is needed.
Blood Volume Replacement
Albumin is the most abundant protein found in blood plasma. Its main function is to maintain the colloidal osmotic pressure of blood in the body. Under certain conditions where the plasma proteins are reduced the blood’s osmotic pressure decreases, resulting in build-up of fluid in the tissues (also known as edema). Edema in specific organs such cerebral and pulmonary edema can prove to be lethal.
Burn victims often loose excessive proteins and are supplemented with albumin as an adjunct to crystalloid therapy. Albumin may also be administered to patients with Adult Respiratory Distress Syndrome (ARDS) where pulmonary edema is accompanied by hypoalbuminemia. Hemorrhages, excessive renal excretion, major injury, malnutrition, surgery and various inflammatory conditions can lead to hypoalbuminemia. A deficit in the albumin levels may be alleviated by infusion with plasma derived human albumin.